BioMarin Pharmaceutical Inc. (BMRN), a leading player in the rare disease space, continues to be optimistic about its future, even in the face of recent competition from Ascendis Pharma A/S (ASND). Last week, Ascendis Pharma released positive topline data from a trial involving children with achondroplasia, a genetic disorder causing dwarfism and disproportionate short stature.
However, Goldman Sachs, after meeting with BioMarin’s leadership, remains confident in the company’s outlook. While acknowledging the potential impact of Ascendis Pharma’s successful trial, Goldman analysts believe BioMarin’s Voxzogo drug will continue to grow. Voxzogo’s broad label, which lacks age restrictions, gives it a competitive advantage over Ascendis Pharma’s potential offering.
Despite the competitive landscape, BioMarin is targeting significant growth. The company expects to achieve approximately $4 billion in total revenue by fiscal year 2027, driven by increased adoption of Voxzogo in the US. BioMarin is also implementing a new business structure, focusing on three key areas: skeletal conditions, enzyme therapies, and Roctavian, its hemophilia A gene therapy. The company is aiming for a low-to-mid 40% adjusted operating margin, starting in 2026, which is being supported by a $500 million cost transformation program.
Goldman Sachs analysts believe BioMarin’s future is bright, with potential for further upside through external business development. While acknowledging the increasing competition, Goldman Sachs maintains a Buy rating with a price target of $139 for BMRN stock.
